NCT03961243

Lentiviral FIX Gene Therapy

Lentiviral FIX Gene Therapy for Hemophilia B

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Shenzhen Geno-Immune Medical Institute · Academic / Other
• Sex: All
• Age: 2 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

Detailed description

Hemophilia B is a genetic bleeding disorder caused by the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding episodes, which can cause chronic joint disease and sometimes even death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of clotting factor concentrates, either prophylactically or in response to bleeding. The procedure is life time long and expensive while still cannot achieve a cure.Gene therapy is a novel technology that has been successfully demonstrated in a number of clinical studies for diseases such as cancer and genetic diseases. In this study, an advanced lentiviral vector system NHP/TYF will be used to deliver a functional FIX gene to overcome human clotting FIX gene defect in patients with hemophilia B. This study is a Phase I trial evaluating the safety and efficacy for infusion of gene modified autologous stem cells in patients with hemophilia B.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
BIOLOGICAL	YUVA-GT-F901	Lentiviral factor IX gene modified autologous hematopoietic and mesenchymal stem cells

Timeline

• Start date: 2020-06-01
• Primary completion: 2022-05-31
• Completion: 2022-06-01
• First posted: 2019-05-23
• Last updated: 2019-05-23